A key stage of exploratory drug development is implementing a proof-of-concept study to demonstrate the safety of a drug. Given the importance of accurate dose-finding for Phase 3 success, methodological improvements to proof-of-concept studies in Phase 2 can translate into greater likelihood of getting a drug to market.
As the diagram above illustrates, however, drugs in nearly all therapeutic areas perform worse in Phase 2 than in other phases of drug development. For a variety of reasons oncology drugs perform exceptionally poorly. As a result, improved methodology in proof-of-concept studies is critical for more successful oncology trials.
In a recent presentation before the Cytel East User Group Meeting, Ashwin Gollerkeri, MD, of Pfizer, demonstrated how patient selection strategies can play a key role in improved proof-of-concept studies in oncology. Using data from Pfizer’s recent experience with NSCLC, Gollerkeri showed how biomarkers were used to identify the appropriate target population for a new drug.Two important conclusions may be drawn from Gollerkeri's presentation:
- The eNPV (expected net present value) of a clinical trial can increase substantially with biomarker enriched populations. By identifying sub-populations in Phase 2 that may be particularly well suited to benefit from a new treatment, pharmaceutical companies can improve the likelihood of bringing a new drug to market.
- Gollerkeri also showed that while the eNPV of a drug can increase with biomarker enriched populations, there are significant benefits to eNPV when biomarker based patient selection is used prospectively rather than retrospectively. As a result, it is important to engage in prospective planning for biomarker enrichment. Cytel Consulting would be happy to assist you in exploring this possibility.
For access to Ashwin Gollerkeri’s presentation slides, click the button below. We will soon be making videos of the presentation (and all other EUGM presentations) available on the Cytel East User Group on LinkedIn.
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